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CNS & Neurological Disorders - Drug Targets
(Formerly Current Drug Targets - CNS & Neurological Disorders)
ISSN (Print): 1871-5273
ISSN (Online): 1996-3181
VOLUME: 9
ISSUE: 6
DOI: 10.2174/187152710793237494      Price:  $58









Genetic Determinants of Amyotrophic Lateral Sclerosis as Therapeutic Targets

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Author(s): Daryl A. Bosco and John E. Landers
Pages 779-790 (12)
Abstract:
Amyotrophic lateral sclerosis (ALS) is an incurable disease resulting from the deterioration of motor neurons. The onset of disease typically occurs in the fifth decade of life and progresses rapidly; death occurs for 75% of patients within 5 years. The only drug that is available to treat ALS is riluzole, which extends survival by just 2-3 months. Thus, new therapeutic directions are being sought to prolong the lifespan of ALS patients. Since the discovery of SOD1 as a genetic determinant of ALS in 1993, SOD1-models of ALS have been extensively employed for the development of ALS therapeutics. Novel genetic targets are now under investigation following the recent discoveries linking TDP-43, FUS/TLS, angiogenin, KIFAP3 and UNC13A to ALS. In this review, we present several of the genetic contributors to both sporadic and familial forms of ALS and discuss their potential as therapeutic targets for this devastating disease.
Keywords:
Amyotrophic lateral sclerosis, neurodegeneration, therapy, genome-wide association, SOD1, TDP-43, FUS/TLS, angiogenin, KIFAP3, UNC13A, familial ALS, Alzheimer's disease, Parkinson's disease, Huntington's disease, immunotherapy, FALS, chaperones, arimoclomol, TAR, SALS, frontotemporal lobar degeneration, (GWAS), DPP6
Affiliation:
Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01605, USA.